Taking Part in Clinical Trials

Clinical research in children is essential if we are to find new ways of preventing, diagnosing and treating childhood diseases. It is the means by which we can ensure that children’s medicines and treatments are safe and effective, and how scientific advances can be made into the future.

Successful research relies on children and their families taking part. More information on clinical research and clinical trials is shown below.

Information for Families

Clinical research is medical research that involves people. Clinical research studies are designed to answer specific questions about health and illness. These studies give important information about how well treatments work and how safe they are.

The term ‘treatment’ can mean different types of healthcare, such as:

Medicines or drugs to treat pain, infection, blood pressure or other sicknesses

Vaccines to prevent infections. These include measles, mumps and rubella vaccines.

A clinical trial is an important type of research study where doctors and researchers compare treatments. They carefully study the information they gather so they can find out if the treatments are effective, safe and how best to use them in adults, children and teenagers.

The only way to really know how well a treatment works in people is to carefully study the treatment in people. A new treatment may or may not be better than an existing one and a clinical trial is needed to find out.

To learn more about how effective the treatments we use are, clinical trials investigate:

How well specific treatments work

How safe the treatments are and by finding out if they have any side-effects

Clinical trials are very important for everybody, but it is really important for children and teenagers because many of the treatments we use today have only been studied in adults.

This is because, until recently, children were rarely included in clinical trials in order to protect them. But ironically this means that there is less information about how children respond to some medications, medical devices and tests.

Children and teenagers often have different medical problems than adults, so the treatments may work differently. Also, children and teenagers may react differently to treatments and so could have different side-effects.

Clinical research in children helps us to treat them according to the child’s needs, rather than as small adults.

Taking part in a clinical research study may lead to knowledge that could benefit your child as well as others. The aim of a clinical trial is to improve treatment, and although your child may or may not benefit from the study, children who are treated in the future will benefit from more information on how effective and safe the treatment is.

It is up to you and your child and family if you want to take part and if you or your child choose not to participate, your decision will be respected. You would still receive the same medical care as before.

What is involved in taking part in a clinical research study depends on the type of study and what is being studied.

It may be as simple as allowing researchers to look at your child’s medical records or to use leftover tissue or blood that is collected from your child as part of their routine medical care. For clinical trials, it may involve new medicines or treatments and extra tests such as ECGs, X-rays or MRI scans. Some research studies may ask you to complete a questionnaire or survey.

Before a child can take part in a research study, the researchers must ask the parents or guardians to give their permission – or ‘consent’. The researcher must explain to the child and their parents what is involved in the study and answer any questions that the family might have. Researchers will also explain the study to each child to the level of their understanding and ask them if they are willing to take part: this is called assent. Before giving their assent, the child must show that they understand and want to take part in the research study.

A child or teenager cannot be entered into a trial without their parents or guardians signing forms saying that they agree for their child to take part. Informed consent is learning the facts about a clinical study before deciding to take part.

As part of the information given to your family before taking part in a research study, you will be told the potential risks and benefits of taking part. You will be given time to consider them and to talk them over with your family as well as your doctor or nurse.

The potential risks can include:

That the new treatment, intervention or test may not work

Unpleasant or serious side effects

The need for additional treatments, tests and hospital visits

The potential benefits can include:

Getting closer monitoring and additional testing that may not be part of standard of care

Gaining early access to new treatments that are not yet available to the public

Longer term benefits for your family or other families by helping researchers learn more about childhood diseases and biology

The Principal Investigator is the person at the hospital responsible for the study. This person is a doctor or another healthcare professional that understands your disease or illness and is looking at ways to improve treatment.

Before any clinical research study can start, it must first be approved by an ethics committee. An ethics committee is an independent group of doctors, nurses, researchers and non-medical people. They carefully examine a detailed description of the study to decide whether it is of good quality and fair to ask people to take part in it.

A clinical trial must also get additional permissions before it can start. The Irish Government has given the Health Products Regulatory Authority (HPRA) the job of looking after the safety of clinical trials involving medicines and medical devices. Before a trial involving medicines or a medical device can go ahead, they must also study a detailed description of the trial and give their permission.

During a study, researchers must send in regular reports to the ethics committee and to the HPRA to ensure the safety of people taking part in the study.

Strict rules are in place to protect the rights, safety, well-being and dignity of people taking part in clinical trials. All study participants are monitored carefully throughout the trial and their safety and well-being is everyone’s priority.

Clinical research studies in children are given extra special attention to make sure that they are safe and are conducted according to strict ethical standards. New medicines or treatments will only be tested if there is a strong reason to believe they might be better than the standard of care.

The family and child are free to change their mind and leave a clinical study at any time before it starts, during the study, or during the follow-up period. Participating in research is always voluntary. Even if you decide to participate, you can always withdraw from the study at any time without affecting your relationship with your doctor or nurse.

All the records of your study information will be kept confidential in the same way as your GP or hospital records. The researchers cannot tell anyone that you are taking part in the trial without asking you first. Strict rules are in place to ensure that personal information is kept confidential.

It includes a series of information booklets for children who may be taking part in clinical trials. The materials provide information for different age groups and were developed in a joint collaboration with the NCRC and IPPOSI (Irish Platform for Patient Organisations Science and Industry).